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FIBROCELL SCIENCE, INC. filed this Form 8-K on 01/08/2019
Entire Document
Company Highlights • Medical breakthroughs for rare diseases of the skin and connective tissue Focus on Rare Skin Diseases • Unmet needs with no approved therapies • Significant mortality and morbidity impact on pediatric populations Proprietary Ex-Vivo Gene • Autologous fibroblasts derived from skin are the vehicle to deliver target proteins locally to Modified Fibroblast the site of disease Platform • Extensive experience culturing dermal fibroblasts, including commercial scale • FCX-007 for the treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB) . Completed enrollment target of six patients (seven enrolled) and initiated dosing in Phase 2 . Well tolerated safety and positive early trends noted in pharmacology and wound healing Two Clinical Trial Programs . Held a Type C meeting on Phase 3 clinical trial design, and received guidance from FDA; submitted meeting request to FDA, and Phase 3 protocol to follow in meeting’s briefing package . FCX-013 for the treatment of moderate to severe Localized Scleroderma . IND allowed by FDA, and initiated first investigator site for clinical enrollment • Regulatory advantages including more frequent communications with FDA, eligibility for Multiple FDA Designations Accelerated Approval and Priority Review, and Rolling Review • Two Rare Pediatric Disease Designations to potentially receive Priority Review Vouchers (PRVs) upon market authorization • In-house 13,000 square foot cGMP cell therapy manufacturing facility supporting both FCX- Internal Manufacturing 007 and FCX-013 Infrastructure • Existing capacity to serve the U.S. RDEB market 3